Archive News 2016

From the 18th to the 24th of September the Global Mitochondrial Disease Awareness Week took place. Around the world Global Mitochondrial Disease Awareness Week was marked with educational, fundraising and advocacy efforts designed to raise awareness about mitochondrial disease (mito). Within this framework many events took place in different countries.

Examples of events to raise awareness and funds for research are: walking and biking events, patient information days, symposia with specific topics, fundraising activities, and a special one is the ‘stay in bed day’ event in Australia. One of the more striking activities was that important landmarks in the world were set in green light (see photo of Nijmegen Radboud UMC).

The GMDAW initiative once started in Australia and is now being organized under the flag of IMP. We proudly present this initiative and hope it will find its way to many countries in the world. Curious about what has been and still is being organized in your own country? You can have a look at the GMDAW website: www.gmdaw.org

The list of potentially harmful drugs for mito's has been updated again and adjusted. You can find the 3rd version of the list on this website under the 'mitodisease' button. At the wonderful suggestion of a mitochondrial patient we have also added printbuttons. Although it is still a 6 page document you can print it and take it with you when you visit your docter. Some time ago a French and Dutch translation were added. Other translations may follow. IMP considers excellent information for patients to be crucial in the empowerment of mitochondrial patients. Please do not hesitate to contact us if you have questions or if you believe that the list can be improved: This email address is being protected from spambots. You need JavaScript enabled to view it.

Khondrion, the clinical-stage pharmaceutical company focusing on small molecule therapeutics for mitochondrial diseases, announces today the initiation of the KHENERGY study, a Phase 2 clinical trial with KH176 in patients harboring the m.3243>G mutation in the mitochondrial genome. This mitochondrial DNA mutation is associated with Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like episodes (MELAS syndrome), Mitochondrial Diabetes and Deafness (MIDD syndrome) and mixed phenotypes.

Khondrion's KH176 is an orallly bio-available small molecule developed by Khondrion for the treatment of mitochondrial (-related) diseases. The compound is a member of a new class of Khondrion drugs essential for the control of oxidative and redox pathologies. In December 2015 Khondrion reported that KH176 demonstrated a favorable pharmacokinetic profile and an acceptable safety profile in randomized, placebo-controlled, double blind Phase 1 clinical trials, performed in healthy male volunteers.

Jan Smeitink, Khondrion's CEO and Professor of Mitochondrial Medicine at the Radboud University Medical Center, Nijmegen, The Netherlands, said "With the successful outcome of the KH176 phase I clinical trials and now the initiation of the KHENERGY STUDY in adult patients we have set an important step to accomplish our mission - making a substantial contribution to the development of drugs for patients suffering from mitochondrial (-related) diseases. Based on the anticipated learnings from the KHENERGY study we will be initiating discussions with the EMA to develop pivotal trials to better characterize the potential benefits of KH176 in patients with mitochondrial disease". Khondrion expects to have enrolled all 20 patients in the fourth quarter of this year.

The KHENERGY STUDY will be a single-center, double-blinded, randomized, placebo-controlled 2-way cross over Phase 2 trial. Patients will receive KH176 orally in a twice a day dosing schedule. The primary efficacy endpoint will be objective, quantitive, clinical relevant gait assessments. The study will also explore changes in other measures of clinical relevance and biomarkers associated with mitochondrial functioning.

About Mitochondrial Diseases

Mitochondria, the cell's powerhouses, produce the energy necessary for life. Mitochondrial failure, due to either mutations in the mitochondrial genome or the nuclear DNA, is associated with a broad range of diseases, including orphan diseases of the oxidative phosphorylation system like Leigh disease, MELAS, MIDD and LHON syndromes as well as diseases like Parkinsons'disease. Cellular consequences like abnormal mitochondrial architecture, reactive oxygen species production and alterations in the cellular redox-state are common findings in these releases. Khondrion's drug development strategy is based on counteracting these cellular consequences to stop disease progression and to restore normal cellular function.

About Khondrion

Khondrion is an innovative Dutch biopharmaceutical mitochondrial medicine company focusing on developing small molecule therapeutics for mitochondrial diseases. The potential of several lead compounds to serve as new treatment modalities for mitochondrial disease is currently being explored. Khondrion's KH176 has been granted Orphan Drug Designation (ODD) for Leigh disease and MELAS syndrome in Europe and for all inherited mitochondrial respiratory chain disorders in the USA. Thanks to its strategic partnership with the Radboud Center for Mitochondrial Medicine of the Radboud University Medical Center, Nijmegen, The Netherlands, Khondrion has access to all mitochondrial tools, technologies and expertise. Besides, Khondrion has established collaborations with patient organizations, patient advocacy groups and university research groups around the world as well as with small, medium and large enterprises. Khondrion is a privately held pharmaceutical company, among others supported by the Dutch Foundations Energy4All, Join4Energy, Road4Energy, Tim Foundation, Zeldzame Ziekten Fonds, and National and European Governments. Khondrion has established a strong intellectual property position protecting its emerging product portfolio through the filing of multiple broad patent applications. For more information, please visit www.khondrion.com.

Cautionary Note Regarding Forward-­‐Looking Statements

This press release contains forward-­‐looking statements. All statements, other than statements of historical facts, contained in this press release, including statements regarding our strategy, future operations, future financial position, future revenues, projected costs, prospects, plans, and objectives of management, are forward-looking statements. The words "anticipate", "believe", "estimate", "expect", "intend", "may", "plan", "predict", "project", "target", "potential", "will", "would", "could", "should", "continue", and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Khondrion. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product development, including the uncertainties of clinical success and the timeline for the availability of KH176. While we may elect to update these forward-looking statements at some point in the future, we specifically disclaim any obligation to do so, even if our views change.

Contacts:

Khondrion BV

Prof. dr. Jan Smeitink, CEO

E-mail: This email address is being protected from spambots. You need JavaScript enabled to view it.

On Friday the 27th of May extraordinary events took place in Ottawa and Calgary, Canada and also in Australia. Team MitoCanada partnered with the AMDF in Australia. A team of 25 amazing people aimed at beating 6 endurance records by running on treadmills. Additionally Team MitoOttawa raised awareness at the Ottawa Marathon.

All this to raise awareness and funds for the fight against mitochondrial disease.

Blaine Penny, with the support of his wife Sarah and daughter Julia, started ‘Running for those who can’t’ and co-founded Team MitoCanada. Team MitoCanada has grown into a successful fundraising arm of MitoCanada, with runners and supporters all over the world.

MitoCanada and the AMDF are both members of IMP and as such active in the worldwide battle against mitochondrial disease.

The events were given tremendous response in the media and from the public.

After the 24 hour effort was finished it turned out that not 6 but 7 world records (Guinness Book of Records) were smashed! Thanks to the fantastic teams the fundraising goal is coming closer. Help them come closer to this goal and donate at

ANAIS, ANTOINE AND MICHAEL HAVE BEEN RUNNING FOR MEET IN PARIS AND RIEUMES, FRANCE. WHO ARE THEY?

My name is Anaïs. I am a PhD student in microbiology and fan of science. I really appreciate and encourage the communication about research in science, in order to better inform the public, and especially the patients, about the recent advances, and most of all to give hope by showing that things are progressing. Running is a beautiful metaphor to say that one moves forward. Finally, I chose to run for your organization because I was sensitive to the message you convey: The Run and good humour to talk about serious topics.

Anaïs

I am Antoine and I'm 33. I'm an engineer in the field of renewable energy. I am the father of two young children (a 3-year-old daughter and a son of 10 months) who are my reason for living. Thus, I easily put myself in these families’ shoes. I was particularly touched by their struggles to promote research and ensure that we get interested in those rare diseases. I thought that if even only 10 runners saw my T-shirt and questioned themselves about the MEET, that would be better than nothing (I always look at that of others)!

Antoine

My name is Michael, I'm 44 years old and I have 2 children: David, who is 14, and Karen who is 12 years old. Karen is affected by mitochondrial disease since she was 2 but she gives us a lot of energy everyday. Twice a week I run about 12 km. It helps me going forward. My wife, Emma, met the MEETers on January during the MEET symposium. Together they have organized the stand in Paris for the running event and I thought it was a great idea to support them by, myself, running this very same week end. My challenge was to run a 24 km trail in the city of Rieumes, close to Toulouse, and I made it! I was so proud to be able, by such means, to bring my own contributions and to help the mito-community. Indeed, my wife is part of A.M.Mi. and IMP and she often goes to different meetings abroad that I cannot really understand, but here, I have been able to help and to convey an important message: Mito-patients needs energy, and by running we can share, we can give some of our energy, to promote awareness about mitochondrial diseases!